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Gene
therapy has made important medical advances in recent years. Within a relatively
short time span, it has moved from the conceptual stage to technology
development and laboratory research to clinical translational trials for a
variety of deadly diseases. Although controversial, gene therapy is poised to
become a viable option for a variety of often life-threatening medical
conditions. It could potentially cure illnesses with a single treatment by altering
the underlying cause of disease, defective genes.

The
Cambridge, MA.-based clinical-stage startup AvroBio is developing transformative,
life-changing therapies for rare diseases and cancer by genetically modifying a
patient’s own cells and infuse these back into the patient. Their first
program, AVR-01, is an immunotherapy approach to acute myeloid leukemia (AML). They
extract the patient’s cancer cells, and modifies it to express a powerful
immune signaling agent. When transferred back into the patient, they activate T
cells to attack the remaining cancer in the body.

The startup’s
second program, AVR-02, is targeted at Fabry disease, a rare inherited disorder
that results from the buildup of a particular type of fat in the body’s cells. Patient’s
cells are genetically modified to add a functional copy of the faulty gene that
causes the disease, expressing an enzyme called alpha-galactosidase when
infused back into the patient.

Gene
therapies represent a new paradigm in human health, with the potential to
deliver dramatic disease-modifying effects with long-lasting and durable
impact. This is especially true for 95% of rare diseases, which still have no
approved treatment. AvroBio’s highly innovative therapies offer potentially
life-altering impact following a single infusion, changing the lives of people
worldwide who so far have very few treatment options. For Fabry patients for
example, this could mean no more frequent trips to infusion centers,
significantly improving quality of life. However, it is still early, and both
treatments are currently in Phase 1 trials.

AvroBio was formally launched in February 2016, and in August
announced they had raised $25 million in a Series A
financing co-led by Atlas Venture, Clarus and SV Life Sciences. The funding
will be used to accelerate the development of its programs in Fabry disease and
AML, and to expand their pipeline in both rare diseases and solid and liquid
cancers.